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Trial · NCT03782792

Effisayil™ 1: A Study to Test Spesolimab (BI 655130) in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis

NCT03782792, Phase 2, Completed, Boehringer Ingelheim; results first posted 2022-03-09

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Effisayil™ 1: A Study to Test Spesolimab (BI 655130) in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis. Trials, record NCT03782792. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03782792

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title
Effisayil™ 1: A Study to Test Spesolimab (BI 655130) in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis
NCT number
NCT03782792
Status
Completed
Phases
Phase 2
Enrollment
53 participants, actual
Lead sponsor
Boehringer Ingelheim
Conditions
Generalized Pustular Psoriasis
Interventions
Spesolimab, Placebo
First posted
2018-12-20
Last updated
2025-10-16
Other registries' IDs
eudract:2017-004231-37
Results first posted
2022-03-09
Limitations and caveats
Due to the trial design, a large proportion of patients had received escape medication or non-randomized spesolimab by Week 4, based on worsening, insufficient response, or non-response. This should be taken into account for the interpretation of the results at Week 4. A large proportion of patients in both arms had been treated as non-responders at Week 4, and the true efficacy outcomes for the randomized treatment at this time-point were never observed for the analysis.

Source ClinicalTrials.gov

Registered outcomes16

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
PrimaryProportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1At Week 1.
The Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) relies on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The investigator (or qualified site personnel) scored the erythema, pustules, and scaling of all GPP lesions from 0 to 4. The GPPGA pustulation subscore ranges from 0 to 4 where: 0 = clear; 1. = almost clear; 2. = mild: 3. = moderate; 4. = severe. A lower GPPGA pustulation subscore indicates a better outcome. A GPPGA pustulation subscore of 0 means no visible pustules. The proportion of patients who achieved a GPPGA pustulation subscore of 0 at Week 1 is reported.
SecondaryKey Secondary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 1At Week 1.
GPPGA relied on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The GPPGA total score was calculated by taking the mean of the erythema subscore, pustules subscore and scaling/crusting subscore. The severity of each subscore was assessed using a 5 point scale score ranging from 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score is assigned as follows: * 0, if scores for all three subscores are 0, * 1, if 0 \< mean \< 1.5, * 2, if 1.5 ≤ mean \< 2.5, * 3, if 2.5 ≤ mean \< 3.5, * 4, if mean ≥ 3.5. A lower GPPGA score indicates a better outcome, with 0 being clear and 1 being almost clear. The proportion of patients with a GPPGA score of 0 or 1 at Week 1 is reported.
SecondaryProportion of Patients With a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4At Week 4.
Generalized Pustular Psoriasis Area and Severity Index (GPPASI) provides a numeric scoring for a patient's overall Generalized Pustular Psoriasis (GPP) disease state, ranging from 0 to 72. It is a linear combination of percent of surface area of skin affected by erythema, pustules, and scaling and the severity of erythema, pustules, and scaling (desquamation) over 4 body regions (head, trunk, upper limbs and lower limbs). A higher score indicates a worse disease state, while a score of 0 indicates no disease. GPPASI 75 is based on the percent reduction from baseline, generally summarized as a dichotomous outcome based on achieving over an 75% reduction. Proportion of patients with GPPASI 75 at Week 4 is reported.
SecondaryChange From Baseline in Pain Visual Analog Scale (VAS) Score at Week 4Baseline and at Week 4.
The pain Visual Analogue Scale (VAS) is a participant-administered single-item scale designed to measure skin pain intensity from generalized pustular psoriasis (GPP) using a 100 millimeter (mm) horizontal VAS. Overall severity of participant's skin pain from GPP is indicated by placing a single mark on the horizontal 100 mm scale from 0 mm (no pain) to 100 mm (pain as bad as one can imagine). Change from baseline was calculated by subtracting the VAS score at baseline from the VAS score at Week 4. A negative change indicates an improvement from baseline. Death, any use of escape medication, OL Spesolimab at Day 8, or rescue medication with Spesolimab after Day 8, prior to observing the endpoint was considered to reflect a failure to achieve the endpoint outcome, i.e. non-response (NR). NR is not a missing value but the worst possible outcome of the endpoint. For example, if the achieved data is (NR, NR, NR, NR, NR, NR, 2, 3, 3, 3, 5) then Q1 is NR, median is NR and Q3 is 3.
SecondaryChange From Baseline in Psoriasis Symptom Scale (PSS) Score at Week 4Baseline and at Week 4.
PSS is a 4-item patient-reported outcome instrument that assesses the severity of psoriasis symptoms in moderate to severe psoriasis patients. The symptoms included are: pain, redness, itching, and burning. The symptom severity was assessed using a 5 point scale ranging from 0 to 4 where 0=none, 1=mild, 2=moderate, 3=severe, 4=very severe. The symptom scores are added to an unweighted total score (range: 0 to 16). A lower PSS score indicates a better outcome. Change from baseline =PSS score at Week 4 - PSS score at baseline. Death, any use of escape medication, OL Spesolimab at Day 8, or rescue medication with Spesolimab after Day 8, prior to observing the endpoint was considered to reflect a failure to achieve the endpoint outcome, i.e. non-response (NR). NR is not a missing value but the worst possible outcome of the endpoint. For example, if the achieved data is (NR, NR, NR, NR, NR, NR, 2, 3, 3, 3, 5) then Q1 is NR, median is NR and Q3 is 3.
SecondaryChange From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Score at Week 4Baseline and at Week 4.
The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function. Each items is scored from 0 to 4. Score range is 0 (extreme fatigue)-52 (no fatigue). Change from baseline=FACIT Fatigue score at Week 4- FACIT-Fatigue score at baseline. Death, any use of escape medication, OL Spesolimab at Day 8, or rescue medication with Spesolimab after Day 8, prior to observing the endpoint was considered to reflect a failure to achieve the endpoint outcome, i.e. non-response (NR). NR is not a missing value but the worst possible outcome of the endpoint. For example, if the achieved data is s (NR, NR, NR, NR, NR, NR, 2, 3, 3, 3, 5) then Q1 is NR, median is NR and Q3 is 3.
SecondaryProportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 4At Week 4.
GPPGA relied on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The GPPGA total score is calculated by taking the mean of the erythema subscore, pustules subscore and scaling/crusting subscore. The severity of each subscore was assessed using a 5 point scale score ranging from 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score is assigned as follows: * 0, if scores for all three scores are 0, * 1, if 0 \< mean \< 1.5, * 2, if 1.5 ≤ mean \< 2.5, * 3, if 2.5 ≤ mean \< 3.5, * 4, if mean ≥ 3.5. A lower GPPGA score indicates a better outcome, with 0 being clear and 1 being almost clear. The proportion of participants with a GPPGA score of 0 or 1 at Week 4 is reported.
SecondaryProportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Sub-score of 0 Indicating no Visible Pustules at Week 4At Week 4.
The Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) relies on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The investigator (or qualified site personnel) scores the erythema, pustules, and scaling of all GPP lesions from 0 to 4. The GPPGA pustulation subscore ranges from 0 to 4 where: 0 = clear; 1. = almost clear; 2. = mild: 3. = moderate; 4. = severe. A lower GPPGA pustulation subscore indicates a better outcome. The proportion of patients who achieved a GPPGA pustulation subscore of 0 at Week 1 is reported.
SecondaryProportion of Patients With a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 4At Week 4.
Generalized Pustular Psoriasis Area and Severity Index (GPPASI) provides a numeric scoring for a patient's overall Generalized Pustular Psoriasis (GPP) disease state, ranging from 0 to 72. It is a linear combination of percent of surface area of skin affected by erythema, pustules, and scaling and the severity of erythema, pustules, and scaling (desquamation) over 4 body regions (head, trunk, upper limbs and lower limbs). A higher score indicates a worse disease state, while a score of 0 indicates no disease. GPPASI 50 is based on the percent reduction from baseline, generally summarized as a dichotomous outcome based on achieving over an 50 % reduction. Proportion of patients with GPPASI 50 at Week 4 is reported.
SecondaryPercent Change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) From Baseline at Week 4Baseline and at Week 4.
GPPASI provides a numeric scoring for a patient's overall Generalized Pustular Psoriasis (GPP) disease state, ranging from 0 (no disease) to 72 (worse disease state). It is a linear combination of percent of surface area of skin affected by erythema, pustules, and scaling and the severity of erythema, pustules, and scaling (desquamation) over 4 body regions (head, trunk, upper limbs and lower limbs). %GPPASI change from baseline=(GPPASI at Week 4-GPPASI at baseline) \*100/(GPPASI at baseline). Death, any use of escape medication, OL Spesolimab at Day 8, or rescue medication with Spesolimab after Day 8, prior to observing the endpoint was considered to reflect a failure to achieve the endpoint outcome, i.e. non-response (NR).NR is not a missing value but the worst possible outcome of the endpoint. For example, if the achieved data is (NR, NR, NR, NR, NR, NR, 2, 3, 3, 3, 5) then Q1 is NR, median is NR and Q3 is 3. Planned statistical analysis were not performed due to lack of valid data.

Outcomes16

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

TypeOutcomeTime framePopulationUnitsMeasured as
PRIMARYProportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1At Week 1.
Randomized Set (RS) (via estimand EN-NRI): This patient set included all randomized patients. EN = Any assessments after death, or any use of escape medication due to disease worsening (defined as worsening of clinical status or GPP skin and/or systemic symptoms as defined by the investigator) prior to Week 1 were considered to represent a non-response. NRI = Non-response imputation for any missing data.
Proportion of participantsNUMBER
SECONDARYKey Secondary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 1At Week 1.
Randomized Set (RS) (via estimand EN-NRI): This patient set included all randomized patients. EN =Any assessments after death, or any use of escape medication due to disease worsening (defined as worsening of clinical status or GPP skin and/or systemic symptoms as defined by the investigator) prior to Week 1 were considered to represent a non-response. NRI = Non-response imputation for any missing data.
Proportion of participantsNUMBER
SECONDARYProportion of Patients With a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4At Week 4.
Randomized Set (RS) (via estimand EN-NRI): This patient set included all randomized patients. EN = Any assessments after death, or any use of escape medication before or after Day 8, open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8 were considered to represent a non-response. NRI = Non-response imputation for any missing data.
Proportion of participantsNUMBER
SECONDARYChange From Baseline in Pain Visual Analog Scale (VAS) Score at Week 4Baseline and at Week 4.
Randomized Set (RS) (via estimand EN-LOCF): This patient set included all randomized patients. EN = Death or any use of escape medication before or after Day 8, open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8 were considered to represent a non-response and assigned with the worst possible outcomes in rank analysis. LOCF = last observation carried forward imputation for any missing data.
Units on a scaleMEDIAN
SECONDARYChange From Baseline in Psoriasis Symptom Scale (PSS) Score at Week 4Baseline and at Week 4.
Randomized Set (RS) (via estimand EN-LOCF): This patient set included all randomized patients. EN = Any assessments after death, or any use of escape medication before or after Day 8, open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8 were considered to represent a non-response and assigned with the worst possible outcomes in rank analysis. LOCF = last observation carried forward imputation for any missing data.
Units on a scaleMEDIAN
SECONDARYChange From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Score at Week 4Baseline and at Week 4.
Randomized Set (RS) (via estimand EN-LOCF): This patient set included all randomized patients. EN = Any assessments after death, or any use of escape medication before or after Day 8, open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8 were considered to represent a non-response and assigned with the worst possible outcomes in rank analysis. LOCF = last observation carried forward imputation for any missing data.
Units on a scaleMEDIAN
SECONDARYProportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 4At Week 4.
Randomized Set (RS) (via estimand EN-NRI): This patient set included all randomized patients. EN = Any assessments after death, or the use of escape medication (before or after Day 8), or open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8, was considered a failure to achieve the endpoint outcome (i.e. non-response). NRI = Non-response imputation for any missing data.
Proportion of participantsNUMBER
SECONDARYProportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Sub-score of 0 Indicating no Visible Pustules at Week 4At Week 4.
Randomized Set (RS) (via estimand EN-NRI): This patient set included all randomized patients. EN = Any assessments after death, or the use of escape medication (before or after Day 8), or open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8, was considered a failure to achieve the endpoint outcome (i.e. non-response). NRI = Non-response imputation for any missing data.
Proportion of participantsNUMBER
SECONDARYProportion of Patients With a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 4At Week 4.
Randomized Set (RS) (via estimand EN-NRI): This patient set included all randomized patients. EN = Any assessments after death, or the use of escape medication (before or after Day 8), or open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8, was considered a failure to achieve the endpoint outcome (i.e. non-response). NRI = Non-response imputation for any missing data.
Proportion of participantsNUMBER
SECONDARYPercent Change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) From Baseline at Week 4Baseline and at Week 4.
Randomized Set (RS) (via estimand EN-LOCF): This patient set included all randomized patients. EN = Any assessments after death, or any use of escape medication before or after Day 8, open-label spesolimab at Day 8, or rescue medication with spesolimab after Day 8 were considered to represent a non-response and assigned with the worst possible outcomes in rank analysis. LOCF = last observation carried forward imputation for any missing data.
Percentage changeMEDIAN

Results by group32

Each measurement the trial posted, group by group, in the words and numbers it posted.

OutcomeGroupCategoryValueSpreadUnits
Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1Placebo0.05695% Confidence Interval 0.010 to 0.258Proportion of participants
Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1Spesolimab 900 mg i.v SD0.54395% Confidence Interval 0.382 to 0.695Proportion of participants
Key Secondary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 1Placebo0.11195% Confidence Interval 0.031 to 0.328Proportion of participants
Key Secondary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 1Spesolimab 900 mg i.v SD0.42995% Confidence Interval 0.280 to 0.591Proportion of participants
Proportion of Patients With a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4Placebo0.11195% Confidence Interval 0.031 to 0.328Proportion of participants
Proportion of Patients With a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4Spesolimab 900 mg i.v SD0.45795% Confidence Interval 0.305 to 0.618Proportion of participants
Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 4PlaceboNAInter-Quartile Range NA to NAUnits on a scale
Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 4Spesolimab 900 mg i.v SD-22.45Inter-Quartile Range -70.41 to NAUnits on a scale
Change From Baseline in Psoriasis Symptom Scale (PSS) Score at Week 4PlaceboNAInter-Quartile Range NA to NAUnits on a scale
Change From Baseline in Psoriasis Symptom Scale (PSS) Score at Week 4Spesolimab 900 mg i.v SD-2.0Inter-Quartile Range -9.0 to NAUnits on a scale

Participant flow10

How many started, completed or left each group, period by period.

PeriodMilestoneGroupParticipantsUnits counted
Overall StudySTARTEDPlacebo18
Overall StudySTARTEDSpesolimab 900 mg i.v SD35
Overall StudyReceived Open Label Treatment With Spesolimab at Wk1/D8Placebo15
Overall StudyReceived Open Label Treatment With Spesolimab at Wk1/D8Spesolimab 900 mg i.v SD12
Overall StudyReceived Rescue Treatment With Spesolimab After Wk1Placebo2
Overall StudyReceived Rescue Treatment With Spesolimab After Wk1Spesolimab 900 mg i.v SD4
Overall StudyCOMPLETEDPlacebo17
Overall StudyCOMPLETEDSpesolimab 900 mg i.v SD32
Overall StudyNOT COMPLETEDPlacebo1
Overall StudyNOT COMPLETEDSpesolimab 900 mg i.v SD3

Adverse events280

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

TypeEventOrgan systemGroupAffectedAt riskEvents
otherAnaemiaBlood and lymphatic system disordersPlacebo118
otherAnaemiaBlood and lymphatic system disordersSpesolimab 900 mg i.v. SD235
otherAnaemiaBlood and lymphatic system disordersOpen Label (OL) D8 Spesolimab 900 mg i.v.027
otherAnaemiaBlood and lymphatic system disordersRescue Spesolimab 900 mg i.v.06
otherErythropeniaBlood and lymphatic system disordersPlacebo118
otherErythropeniaBlood and lymphatic system disordersSpesolimab 900 mg i.v. SD035
otherErythropeniaBlood and lymphatic system disordersOpen Label (OL) D8 Spesolimab 900 mg i.v.027
otherErythropeniaBlood and lymphatic system disordersRescue Spesolimab 900 mg i.v.06
otherPalpitationsCardiac disordersPlacebo118
otherPalpitationsCardiac disordersSpesolimab 900 mg i.v. SD035

Adverse event totals12

The totals the trial posted for each group.

TotalGroupAffectedAt risk
Total, all-cause mortalityPlacebo018
Total, all-cause mortalitySpesolimab 900 mg i.v. SD035
Total, all-cause mortalityOpen Label (OL) D8 Spesolimab 900 mg i.v.027
Total, all-cause mortalityRescue Spesolimab 900 mg i.v.06
Total, other adverse eventsPlacebo1318
Total, other adverse eventsSpesolimab 900 mg i.v. SD2335
Total, other adverse eventsOpen Label (OL) D8 Spesolimab 900 mg i.v.1427
Total, other adverse eventsRescue Spesolimab 900 mg i.v.46
Total, serious adverse eventsPlacebo318
Total, serious adverse eventsSpesolimab 900 mg i.v. SD635

Papers4

The papers the trial lists on ClinicalTrials.gov.

PMIDListed asSource
33785490DERIVEDPubMed
34936739DERIVEDPubMed
37840119DERIVEDPubMed
39104539DERIVEDPubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

ComparisonSource
Spesolimab 900 mg i.v SD vs Placebo: Number of Patients With Treatment Emergent Adverse Events (TEAEs) Within the Treatment PhaseTrial comparisons
Spesolimab 900 mg i.v SD vs Placebo: Number of Patients With Treatment Emergent Adverse Events (TEAEs) up to Week 1Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov
2026-11-12
ClinicalTrials.gov
Results, from AACT
2026-03-23
AACT
NIH grant links
2026-11-13
NIH RePORTER

Sources 3

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
  • NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ClinicalTrials.govnone stated
  • NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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